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7h ago

Global Regulators Halt Elevidys Gene Therapy Over Safety Concerns

Global regulators have paused Elevidys shipments over acute liver failure concerns leaving families in limbo with no active market for the treatment.

Cayse Blair, who has Duchenne muscular dystrophy, was supposed to be dosed with a gene therapy this summer before the FDA asked it be pulled from the market.
MIAMI, FL - MARCH 23: Bottles of antidepressant pills Wellbutrin (L-R) , Paxil, Lexapro, Effexor, Zoloft and Fluoxetine are shown March 23, 2004 photographed in Miami, Florida. The Food and Drug Administration asked makers of popular antidepressants to add or strengthen suicide-related warnings on their labels as well as the possibility of worsening depression especially at the beginning of treatment or when the doses are increased or decreased. (Photo Illustration by Joe Raedle/Getty Images)
A sign marks the offices of Sarepta Therapeutics in Cambridge, Massachusetts, U.S., July 22, 2025. REUTERS/Brian Snyder/File Photo
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Overview

  • On July 18 the FDA requested a voluntary pause of Elevidys shipments in the U.S., which Sarepta agreed to on July 21 after initial resistance.
  • The European Medicines Agency issued a negative opinion on July 25 and health authorities in Japan and Brazil suspended distribution, leaving the therapy on global hold.
  • Elevidys gained accelerated FDA approval in June 2023 and expanded approval in June 2024 despite failing to demonstrate improvement in key clinical outcomes.
  • Two teenage boys with Duchenne muscular dystrophy died from acute liver failure after receiving Elevidys, and more than 900 patients have been treated to date.
  • Sarepta has cut 36% of its workforce and is working with regulators on safety measures as families face cancelled infusion appointments.